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Inavolisib

New Drug Approvals

3] Inavolisib was approved for medical use in the United States in October 2024. [3] 8] Therefore, it may serve as a new addition to combination therapy with conventional cancer treatment, such as chemotherapy. 3] [6] [20] [21] The drug application was granted priority review and breakthrough therapy designations by the FDA. [3]

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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52
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Pirtobrutinib

New Drug Approvals

7] Pirtobrutinib was approved for medical use in the United States in January 2023, [4] [8] [9] [10] and in the European Union in November 2023. [2] 4] Participants had a median of three prior lines of therapy, with 93% having two or more prior lines. [4] 5] It is taken by mouth. [1] 2] PATENTS Guisot, N. 27 January 2023. CCR-23-1272.

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Taletrectinib

New Drug Approvals

1] [2] Adverse effects The FDA prescribing information for taletrectinib includes warnings and precautions for hepatotoxicity, interstitial lung disease/pneumonitis, QTc interval prolongation, hyperuricemia, myalgia with creatine phosphokinase elevation, skeletal fractures, and embryo-fetal toxicity. [1] Food and Drug Administration (FDA).

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Crinecerfont

New Drug Approvals

2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 2] The FDA granted the approval of Crenessity to Neurocrine Biosciences, Inc. [2] Food and Drug Administration (FDA) (Press release).

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Levers for Biological Progress

Codon

More specifically, it reveals that the price to sequence a human genome fell from $100 million in 2001 to $700 by 2021; a stunning collapse in price. Today, a single injection of an FDA-approved gene therapy, called Hemgenix , cures this disease. The cost of sequencing a human genome.

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Landiolol

New Drug Approvals

Landiolol 133242-30-5 ONO-1101 Ono 1101 WHO 7516 FDA APPROVED 11/22/2024, Rapiblyk , To treat supraventricular tachycardia C25H39N3O8 509.6 28 October 2021. FDA Approves AOP Health’s Rapiblyk (landiolol) for Atrial Fibrillation and Atrial Flutter in the Critical Care Setting” (Press release).

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